Lower-dose ATG plus mini post‑transplant cyclophosphamide to prevent GVHD after haploidentical stem cell transplant

Reduced-dose Anti-thymocyte Globulin Plus Mini-dose Post-transplant Cyclophosphamide for GVHD Prevention in Haploidentical Donor HCT for Hematologic Malignancy

This approach tries a lower dose of ATG combined with a short course of post‑transplant cyclophosphamide to prevent graft‑versus‑host disease in people aged 12–65 receiving a haploidentical stem cell transplant for acute leukemia or myelodysplastic syndrome.

Quick facts

• Phase: Phase 2
• Study type: Interventional
• Enrollment: 40 (estimated)
• Ages: 12 Years to 65 Years
• Sex: All
• Sponsor: Peking University People's Hospital (Academic / other)
• Drugs / interventions: cyclophosphamide
• Locations: 1 site (Beijing)
• Trial ID: NCT06984536 on ClinicalTrials.gov

What this trial studies

This is a Phase 2 interventional protocol testing a reduced dose of anti‑thymocyte globulin (ATG) together with a mini course of post‑transplant cyclophosphamide (PTCy) as graft‑versus‑host disease (GVHD) prophylaxis after haploidentical hematopoietic stem cell transplantation. Eligible patients are 12–65 years old undergoing their first haploidentical transplant for acute leukemia in remission or myelodysplastic syndrome, excluding maternal and collateral donors. The regimen aims to balance immune suppression to lower GVHD risk while limiting infection and relapse, with close monitoring of transplant‑related mortality, infections, GVHD incidence, and disease control. The single‑center study is conducted at Peking University People's Hospital with planned follow‑up to track safety and efficacy outcomes.

Who should consider this trial

Good fit: People aged 12–65 with acute leukemia in remission or myelodysplastic syndrome who are scheduled for their first haploidentical donor transplant (not from maternal or collateral donors) with ECOG ≤3 and acceptable organ function are ideal candidates.

Not a fit: Patients with refractory disease, severe organ dysfunction, uncontrolled active infection, other untreated malignancies, expected survival under three months, a history of severe allergic reactions, or who are pregnant or breastfeeding are unlikely to qualify or benefit.

Why it matters

Potential benefit: If successful, this regimen could reduce severe GVHD and transplant‑related complications, improving survival and quality of life after haploidentical transplantation.

How similar studies have performed: Established haploidentical approaches using ATG (Beijing protocol) or full-dose PTCy (Baltimore protocol) have been successful, but combining reduced ATG with mini‑PTCy is a newer strategy with limited prior data.

Eligibility criteria

Inclusion Criteria:

1. Patients with AL - CR and/or myelodysplastic syndromes undergoing allogeneic hematopoietic stem cell transplantation for the first time;
2. No gender limit, aged 12 - 65 years;
3. Planned haploidentical donor transplantation, excluding transplantation from maternal and collateral donors;
4. Eastern Cooperative Oncology Group (ECOG) performance status score≤3 points;
5. Baseline organ function tests meet the following criteria:

(1) Left ventricular ejection fraction (LVEF) \> 55%; (2) Serum creatinine ≤ 1.5 × upper limit of normal (ULN).

Exclusion Criteria:

1. Patients with severe dysfunction of brain, heart, kidney or liver;
2. Those in refractory malignant status;
3. Patients with other malignancies requiring treatment;
4. Presence of uncontrolled severe active infection clinically;
5. Expected survival period of less than 3 months;
6. History of severe allergic reactions;
7. Pregnant or breastfeeding women; (8)Presence of any condition deemed by the investigator as unsuitable for study enrollment.

Where this trial is running

Beijing

• Peking University People'S Hospital — Beijing, China (Recruiting)

Study contacts

• Study coordinator: Yu Wang, M.D.

Conditions Myelodysplastic Syndrome; Acute Leukemia